Genzyme Pompe Program Update- November 17, 2008
We would like to take this opportunity to provide an important update to the Pompe
The US Food and Drug Administration (FDA) has informed Genzyme that the FDA
plans an accelerated approval for alglucosidase alfa produced at the 2000 L bioreactor
scale for the treatment of late onset Pompe disease. Prior to approval, the company and
the agency first need to agree on the design of a post-approval verification study and the
FDA must complete its review of the Risk Evaluation and Mitigation Strategy (REMS),
which Genzyme submitted earlier this month. This REMS program is intended to ensure
that distribution of the 2000 L product is prescribed and administered to the intended
patient population. The FDA has classified this submission as a major amendment to the
Biologics License Application (BLA) for alglucosidase alfa produced at the 2000 L scale.
Therefore, to provide time to agree on the design of a post-approval verification
study and complete the review of the REMS submission, the FDA has changed the
Prescription Drug User Fee Action (PDUFA) date to February 28, 2009. Genzyme
will be required to submit the final protocol for the verification study after approval.
Genzyme is pleased to have clarity from the FDA on what it will take for alglucosidase
alfa produced at the 2000 L to be approved under an accelerated approval. An
accelerated approval is not a priority or fast-track review. An accelerated approval is an
approval for commercial marketing, but with the requirement to conduct further studies
to verify clinical benefit. We will work closely with the FDA to design a post-approval
verification study.
Due to our continued state of limited supply, regrettably, MTAP will remain closed to
new adult patients throughout this 3 month extension. Upon approval of alglucosidase
alfa produced at the 2000 L scale, it is anticipated that new adult patients with late-onset
Pompe disease will be able to access product through regular commercial channels.
Patients currently enrolled in MTAP can continue in MTAP until the 2000 L scale is
approved, at which time we will work to transition MTAP participants to commercial
therapy. Alglucosidase alfa produced at the 160 L scale continues to be commercially
available for all patients 17 years of age and younger.
Genzyme recognizes the difficulty this delay may cause for some Pompe patients and
their families, as well as the physicians managing their care and appreciate your ongoing
patience and understanding.